Clinical trial 'immunosuppressive treatment in Duchenne muscular dystrophy' – update

In various controlled trials treatment with corticosteroids has been shown to be effective in patients with Duchenne muscular dystrophy, but their long-term use is limited by severe side-effects. Due to these side-effects therapists have been forced to use intermittent treatment regimens with corticosteroids which produce less side-effects but might also be less effective. Cyclosporin is an immunosuppressive drug already used as a corticosteroid sparing agent for a variety of disorders in childhood. Cyclosporin A was the only immunosuppressant non-steroidal drug to have shown effectiveness in Duchenne muscular dystrophy.

This multicentre, randomised, placebo-controlled trial consists of two phases and includes patients with Duchenne muscular dystrophy from 5 years until loss of independent ambulation. In Phase I the short-term efficiency of cyclosporin (3–4mg/kg/d) as compared to placebo is studied over a period of three months. In Phase II all patients receive intermittent therapy with prednisolone (0.75mg/kg/d; 10 days on –10 days off) in combination with either cyclosporin or placebo over a period of 12 months. The primary outcome is the change in global strength by manual muscle testing (modified MRC scale). Secondary outcomes include myometry of selected muscle groups, timed functional tests, and standardised quality of life questionnaires.

At 11 studycenters in Germany, Austria and Switzerland a total number of 150 patients will be included in this clinical trial.

At the current state over 40 patients are included in this clinical trial. Serious adverse events were not presented and the medication was tolerated by al patients.